Clinical Trial Details

NCT ID: NCT02595489
Date Last Changed: October 12, 2016

Overview

Research Study Summary

A Phase 1/Phase 2 clinical study for patients with X-linked Adrenoleukodystrophy

Research Study Title

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

Purpose

In this pilot study, the investigators will assess the safety of two high-dose regimens of oral vitamin D supplementation and measure the effects of vitamin D supplementation on markers of oxidative stress and inflammation in the blood and brain of study participants before, during, and after taking vitamin D supplements.

The goal of the study is to establish research measures (i.e. biomarkers) and an optimal dose for vitamin D supplementation in boys with the X-linked adrenoleukodystrophy (ALD) genotype.

To Learn more

Recruitment Details

Phase
1/2
Gender
Male
Age
3 to 12 Years
Overall Status
Recruiting
Lead Sponsor
Stanford University
Duration
36 Months
Facility Type
N/A
Compensation

Eligibility

Male ages 3 Years to 12 Years

Criteria for enrollment to screening:

  1. Molecular confirmation of X-linked ALD (VLCFA elevation & ABCD1 mutation)

  2. Male

  3. Age 3yrs - 12yrs at screening

Criteria for assignment to drug:

  1. Plasma 25-hydroxy vitamin D level ≤ 35ng/ml in past 90 days

  2. MRI brain in past 30 days that is negative for evidence of active cerebral demyelination

Exclusion Criteria:

  • history of liver or kidney disease

  • history of nephrolithiasis

  • history of hyperthyroidism

  • history of ulcerative colitis, Crohn's disease, celiac disease

  • taking medication interfering with gastrointestinal absorption

  • contraindication or inability to complete MRI every 6 months

Site Locations (1)

Country State City Zip Facility and Contact
United States California Palo Alto 94304 Stanford University
Keith Van Haren, MD
kpv@stanford.edu

Keith Van Haren, MD
Principal Investigator

Contact

Keith Van Haren, MD

E-mail:

DISCLAIMER: CenterWatch does not conduct clinical research. CenterWatch is a publishing company that posts clinical trials information on behalf of sponsor companies, contract research organizations, clinical research sites and other interested parties. This information is designed to help patients find clinical trials of interest and contact the research centers conducting the trials.